期刊
MOVEMENT DISORDERS
卷 22, 期 13, 页码 1962-1964出版社
WILEY-LISS
DOI: 10.1002/mds.21632
关键词
huntington's disease; histone deacetylase inhibitor; sodium phenylbutyrate
资金
- NCRR NIH HHS [5 M01 RR000334] Funding Source: Medline
Transcriptional dysregulation in Huntington's disease (HD) is mediated in part by aberrant patterns of histone acetylation. We performed a dose-finding study in human HD of sodium phenylbutyrate (SPB), a histone deacetylase inhibitor that ameliorates the HD phenotype in animal models. We used a dose-escalation/de-escalation design, using prespecified toxicity criteria and standard clinical and laboratory safety measures. The maximum tolerated dose was 15 g/day. At higher doses, toxicity included vomiting, lightheadedness, confusion, and gait instability. We saw no significant laboratory or electrocardiographic abnormalities. Gene expression changes in blood suggested an inverse dose-response. In conclusion, SPB at 12 to 15 g/day appears to be safe and well-tolerated in human HD. (C) 2007 Movement Disorder Society.
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